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Jose-Alein Sahel

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Open access Aug 2026

Reprogramming translation for rare disease therapy: challenges posed by large genes

This study offers the most compelling clinical evidence so far that gene replacement therapy can safely restore signi fi cant hearing in children with OTOF-related deafness, with bene fi ts lasting up to 2.5 years, setting a new standard for treating inherited sensory disorders.

B. Pattnaik, Jose-Alein Sahel · 0 citations